Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
Study Details
Study Description
Brief Summary
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease. In addition, investigators apply the Cost-effectiveness analysis (CEA) as a form of economic analysis that compares the relative costs and outcomes (effects) for different treatment regimens on vaso-occlusive painful crisis.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Detailed Description
"Sickle cell disease is an inherited blood disorder characterized by defective hemoglobin (a protein in red blood cells that carries oxygen to the tissues of the body).
Sickle cell disease involves the red blood cells, or hemoglobin, and their ability to carry oxygen. Normal hemoglobin cells are smooth, round, and flexible, like the letter "O," so they can move through the vessels in our bodies easily. Sickle cell hemoglobin cells are stiff and sticky and form into the shape of a sickle, or the letter "C," when they lose their oxygen. These sickle cells tend to cluster together and cannot easily move through the blood vessels. The cluster causes a blockage in small arteries or capillaries and stops the movement of healthy, normal oxygen-carrying blood. This blockage is what causes the painful and damaging complications of sickle cell disease".
"Acute vaso-occlusive crisis (VOC) is a hallmark of sickle cell disease (SCD). Multiple complex pathophysiological processes can result in pain during a VOC. Despite significant improvements in the understanding and management of SCD, little progress has been made in the management of pain in SCD, although new treatments are being explored".
The Painful Episodes:
"The day-to-day management of sickle cell disease often equates with the management of acute and chronic pain. Patients manage many painful events at home so that hospital visits underestimate the frequency of pain
Acute painful episodes are the most commonly encountered vaso-occlusive events in patients of all ages. Presumed to be caused by sickle vaso-occlusion, pain often starts in young children as the hand-foot syndrome or dactylitis, a painful swelling of hands and feet due to inflammation of the metacarpal and metatarsal periosteum. Painful episodes, which last from hours to many days, usually occur with little warning and a clear precipitating event is not often found.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Omega-3 experimental group 50 patients from each participating hospital that will receive Omega-3 supplementation (300-400mg EPA & 200-300mg DHA) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. |
Drug: Omega 3
Omega-3 supplementation (300-400mg EPA & 200-300mg DHA) per day for 8 consecutive months up to 10 months
Other Names:
Drug: Hydroxy Urea
50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.
Other Names:
Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Names:
Drug: Morphine Sulfate
Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.
Other Names:
Procedure: blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.
|
| Experimental: Vit-D experimental group 50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. |
Drug: Vit D
50 patients from each participating hospital that will receive Vit-D medication (1500 IU to 3500 IU ) per day for 8 consecutive months up to 10 months.
in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.
Other Names:
Drug: Hydroxy Urea
50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.
Other Names:
Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Names:
Drug: Morphine Sulfate
Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.
Other Names:
Procedure: blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.
|
| Experimental: Zinc supplements experimental group 50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. |
Drug: Zinc sulfate
50 patients from each participating hospital that will receive Zinc supplements (15 mg to 50 mg ) per day for 8 consecutive months up to 10 months.
in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.
Other Names:
Drug: Hydroxy Urea
50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.
Other Names:
Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Names:
Drug: Morphine Sulfate
Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.
Other Names:
Procedure: blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.
|
| Experimental: Statin experimental group 50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months. in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved. |
Drug: Statins (Cardiovascular Agents)
50 patients from each participating hospital that will receive Simvastatin orally (20 mg to 40 mg ) per day for 8 consecutive months up to 10 months.
in addition to the experimental treatment, this group will receive the traditional treatment of hydroxyurea, Folic acid, pain killer plus regular blood transfusion with a dose de-escalation methods till efficacy of experimental treatment proved.
Other Names:
Drug: Hydroxy Urea
50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.
Other Names:
Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Names:
Drug: Morphine Sulfate
Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.
Other Names:
Procedure: blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.
|
| Active Comparator: Ordinary hospital treatment group 50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months. in addition, Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response in addition, Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose. This group received regular blood transfusion session. |
Drug: Hydroxy Urea
50 patients from each participating hospital that will receive the ordinary treatment of Hydroxyurea (20 mg/kg/day) with monitoring blood count every 2 weeks maximum daily dose: (40 mg/kg/day) for 8 consecutive months up to 10 months.
Other Names:
Drug: Folic Acid Supplementation
Folic Acid dose of 0.5 to 1 mg daily for 3 to 4 weeks until definite hematologic response
Other Names:
Drug: Morphine Sulfate
Morphine medication as a pain killer is administered, if Patient weight <50 kg: Opioid naïve: Initial: 0.05 mg/kg/dose; usual maximum initial dose: 1 to 2 mg/dose.
Other Names:
Procedure: blood transfusion session
Regular blood transfusion session based on patient hematological profile starts from one session every 2 weeks.
|
Outcome Measures
Primary Outcome Measures
- C-reactive protein mg/L [10 months]
C-reactive protein milligrams per deciliter
- Hematocrit % [10 months]
Hematocrit level in percentage value
- Fibrinogen mg/dl [10 months]
Fibrinogen concentration in milligrams per deciliter
- Total cholesterol Mg/dl [10 months]
Total cholesterol milligrams per deciliter
- HDL cholesterol Mg/dl [10 months]
HDL cholesterol milligrams per deciliter
- LDL cholesterol Mg/dl [10 months]
LDL cholesterol milligrams per deciliter
- Triglycerides Mg/dl [10 months]
Triglycerides milligrams per deciliter
- leukocytes count μl [10 months]
leukocytes in microliter
- hemoglobin (Hbg) g/dL [10 months]
hemoglobin (Hbg) gram/deciliter
- White blood cells count [10 months]
White blood cells count in a cubic milliliter of blood
- Lactic acid dehydrogenase U/L [10 months]
Lactic acid dehydrogenase unit per litter
- Reticulocyte count % [10 months]
Reticulocyte count percentage
- Red blood cell (erythrocyte ) sedimentation rate mm/hr [10 months]
erythrocyte sedimentation rate in millimeters (mm) per one hour(hr)
- lymphocyte count µL [10 months]
lymphocyte count in 1 microliter (µL) of blood
- Granulocyte absolute count cells/microliter [10 months]
Granulocyte cells numbers in microliter
- Granulocytes,percentage (GR, pct) [10 months]
percentage of white blood cells with granules in percentage
Eligibility Criteria
Criteria
Inclusion Criteria:
Any case with the full manifestation of sickle cell disease accompanied by acute painful crisis aged from 5-15 years old.
Exclusion Criteria:
-
The presence of any other chronic illness.
-
Patient age>18 years old or < 3 years old.
-
Patients with hepatic diseases including cholestasis hepatic encephalopathy and jaundice.
-
Patients with renal impairment
-
Diabetic patients
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Faculty of medicine, Beni-suef univeristy - Beni-Seuf university hospital | Banī Suwayf | Egypt | ||
| 2 | Faculty of Pharmacy, Beni-Suef university | Banī Suwayf | Egypt | ||
| 3 | Health insurance hospital | Banī Suwayf | Egypt | ||
| 4 | Maternity and Children hospital | Mecca | Saudi Arabia |
Sponsors and Collaborators
- Beni-Suef University
- Benisuef university hospital
- University of Arizona
- Maternity and Children Hospital, Makkah
Investigators
- Study Director: John E. Murphy [Professor of Pharmacy Practice and Science], PharmD, University of Arizona, College of Pharmacy
- Study Director: Mohamed H Meabad [Prof of Pediatrics], M.D, Beni-Suef University, Faculty of medicine
- Study Director: AHMED A ALBERRY [Assistant prof of clinical pharmacology], M.D, Beni-Suef University, Faculty of medicine
- Study Director: RAGHDA R SAYED [Lecturer of Clinical Pharmacy, Ph.D., Beni-Suef University, Faculty of Pharmacy
- Principal Investigator: Shaimaa M Nashat Sayed Abdelhalim, Ph.D Student, Beni-Suef University, Faculty of Pharmacy
- Study Director: Ahmed F Mahmoud Hussein, MS.c, Beni-Suef Health insurance hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- Novel TTT of pedia VOC/SCA